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01-11-2004 | Original Research Article

The Impact of Unlicensed and Off-Label Drug Use on Adverse Drug Reactions in Paediatric Patients

Authors: Dr Antje Neubert, Harald Dormann, Jutta Weiss, Tobias Egger, Manfred Criegee-Rieck, Wolfgang Rascher, Kay Brune, Burkhard Hinz

Published in: Drug Safety | Issue 13/2004

Abstract

Background and objective: Many drugs that are used to treat children are either not licensed for use in paediatric patients (unlicensed) or prescribed outside the terms of the product licence (off label). The incidence of adverse drug reactions (ADRs) associated with the use of such drugs is yet to be established. This study investigates, for the first time in a German patient population, the impact of unlicensed and off-label drug use on ADRs in paediatric patients.

Patients and methods: An 8-month prospective pharmacoepidemiological cohort-based survey was conducted on a ten-bed paediatric isolation ward at the University Hospital Erlangen-Nüremberg, Germany. All patients were intensively monitored for ADRs by a pharmacoepidemiological team. ADRs were characterised according to international classification methods. All drug prescriptions were evaluated retrospectively as to unlicensed or off-label use on the basis of the product information.

Results: A total of 178 patients were included in the study and 740 drug prescriptions were given to 156 patients (median three prescriptions per patient). In 198 cases (27.7% of all prescriptions) drugs were used in either an unlicensed (n = 3) or off-label (n = 195) manner. A total of 46 ADRs were observed in 31 patients (17.4%). Patients receiving at least one unlicensed or off-label drug prescription during hospitalisation (n = 92) experienced an ADR significantly more frequently (n = 26 patients) than patients receiving only licensed drugs (n = 64 vs 5 patients). ADRs were associated with 29 (5.6%) of the 517 licensed drug prescriptions and with 12 (6.1%) of the 198 unlicensed or off-label drug prescriptions. The majority of ADRs caused by unlicensed and off-label drug use were recognised by the attending physician. However, statistical analysis revealed no significant difference in the number of licensed and unlicensed/off-label drug prescriptions causing ADRs.

Conclusion: This study demonstrated that at a paediatric isolation ward the incidence of ADRs caused by unlicensed or off-label drug use was not significantly more than that caused by the licensed drug use. However, patients treated with unlicensed or off-label drugs were shown to possess a significantly increased risk for developing ADRs.

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Title: The Impact of Unlicensed and Off-Label Drug Use on Adverse Drug Reactions in Paediatric Patients
Authors: Dr Antje Neubert
Harald Dormann
Jutta Weiss
Tobias Egger
Manfred Criegee-Rieck
Wolfgang Rascher
Kay Brune
Burkhard Hinz
Publication date: 01-11-2004
Publisher: Springer International Publishing
Published in: Drug Safety / Issue 13/2004
Print ISSN: 0114-5916
Electronic ISSN: 1179-1942
DOI: https://doi.org/10.2165/00002018-200427130-00006

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The Impact of Unlicensed and Off-Label Drug Use on Adverse Drug Reactions in Paediatric Patients

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